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Witness Appearing Before the
Senate Subcommittee on Labor-HHS-Education Appropriations

Fiscal Year 2005 Budget Request

Anthony S. Fauci, M.D.
Director
National Institute of Allergy and Infectious Diseases
National Institutes of Health

April 1, 2004

William Beldon, Acting Deputy Assistant Secretary, Budget 


Mr. Chairman and Members of the Committee:

I am pleased to present the President's budget request for the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH). The Fiscal Year (FY) 2005 budget of $4,425,507,000 includes an increase of $122,467,000 over the FY 2004 enacted level of $4,303,040,000, comparable for transfers proposed in the President's request.

NIAID conducts and supports research studies to understand, treat, and prevent infectious diseases such as HIV/AIDS and other sexually transmitted infections, influenza, tuberculosis, malaria, and illness from potential agents of bioterrorism. In addition, the Institute supports research on transplantation and immune-related illnesses, including autoimmune disorders, asthma and allergies. For 56 years, NIAID-sponsored research has led to new therapies, vaccines, diagnostic tests, and other technologies that have improved the health of millions of people worldwide. Historically, NIAID has accomplished its mission with a strong commitment to basic and targeted research in immunology, microbiology, and infectious disease, disciplines that are related and complementary. The new initiatives of the NIH Roadmap, and the information, reagents and infrastructure they will produce, will further promote the efficient and effective movement of NIAID discoveries from the laboratory bench to the bedsides of patients.

The NIAID Research Response to the Threat of Bioterrorism

The use of deadly pathogens such as smallpox or anthrax as agents of bioterrorism is a serious threat to the citizens of our nation and the world, and biodefense research to mitigate this threat is a key focus of NIAID research. Since the anthrax attacks of 2001, NIAID has significantly strengthened, accelerated, and expanded our biodefense research program. NIAID-supported biodefense research includes: a) basic studies of the structure, ecology, and disease-causing mechanisms of microbes that could be used by bioterrorists; b) the response of the immune system to these pathogens, and; c) the translation of this knowledge into safe and effective countermeasures -- treatments, diagnostics, and vaccines. To achieve our biodefense research goals, NIAID works closely with partners in academia, industry, and other private and public-sector agencies. Research on potential agents of bioterror promises to enhance not only our preparedness for bioterrorism, but also for naturally occurring endemic and emerging infectious diseases.

Progress in biodefense research has been swift and substantial. More than 50 major NIAID initiatives involving intramural, academic and industrial partners have been undertaken. As part of this effort, the Institute has greatly increased biodefense research capacity. For example, NIAID recently funded eight Regional Centers of Excellence for Biodefense and Emerging Infectious Diseases Research. This nationwide network of multidisciplinary academic centers will conduct wide-ranging research on infectious diseases and the development of diagnostics, therapeutics and vaccines. In addition, NIAID is supporting the construction of two National Biocontainment Laboratories (NBLs) and nine Regional Biocontainment Laboratories (RBLs). These high-level biosafety facilities promise to speed the development of effective therapies, vaccines and diagnostics for diseases caused by agents of bioterror as well as for naturally occurring emerging diseases such as SARS and avian influenza.

In addition, NIAID has developed and expanded contracts to screen new drugs; develop new animal models and establish a reagent and specimen repository. NIAID also has made a significant investment in determining the genetic sequences of the genomes of a range of pathogens, which has helped to illuminate the workings of all classes of microorganisms. NIAID-supported researchers and their international colleagues have sequenced genomes representative of all bacteria considered bioterror threats (including multiple strains of the anthrax bacterium), as well as at least one strain of every potential viral and protozoan bioterror pathogen. NIAID also is funding research to better understand the body's own protective mechanisms. A new NIAID program, the Cooperative Centers for Translational Research on Human Immunology and Biodefense, will conduct research to better understand the human immune response to potential agents of bioterror, with the objective of developing new bioterror countermeasures. Another large-scale program is funding sophisticated studies of the human innate system, comprised of the cells that are the "first responders" to infection. Boosting innate immunity holds great promise for developing fast-acting countermeasures to mitigate the effects of bioterror pathogens or toxins.

The ultimate goal of all NIAID biodefense research is the development of medical countermeasures. NIAID-supported scientists have identified: a) antivirals that may play a role in treating smallpox or the complications of smallpox vaccination; b) several approaches to blocking the toxins of the anthrax bacterium; as well as c) antibiotics, antivirals and antitoxins against other major bioterror threats. New and improved vaccines against smallpox, anthrax and other potential agents also are being developed, with the objective of adding them to the Strategic National Stockpile (SNS). For example, NIAID has sponsored the development of a next-generation anthrax vaccine known as rPA, with the goal of adding 75 million doses to the SNS to protect U.S. citizens. Clinical trials of rPA are ongoing; results to date build on similar findings in animal studies and suggest that the vaccine is safe and capable of evoking a robust immune response. Researchers also will test whether the currently recommended course of antibiotic therapy for individuals exposed to anthrax spores can be reduced by vaccinating exposed subjects with rPA.

NIAID-supported researchers also are testing several new smallpox vaccines that may prove at least as effective as the current smallpox vaccine, but with fewer side effects. One of these, modified vaccinia Ankara (MVA), is based on a strain of the vaccinia virus that replicates less robustly than the traditional Dryvax vaccinia virus, and is known to cause fewer side effects than the latter. Human trials of MVA vaccines are underway at NIH and elsewhere. Encouragingly, recent studies by NIAID intramural scientists and their colleagues have shown that MVA protects monkeys and mice from smallpox-like viruses. NIH also has launched the first human trial of a vaccine designed to prevent infection with Ebola virus. The trial vaccine, a type called a DNA vaccine, is similar to other investigational vaccines that hold promise for controlling such diseases as AIDS, influenza, malaria and hepatitis.

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HIV/AIDS Research

Most recent estimates on the scope of the HIV/AIDS pandemic are profoundly sobering. Approximately 40 million people worldwide are living with HIV/AIDS. In 2003 alone, 5 million people worldwide were newly infected with HIV -- about 14,000 each day, more than 95 percent of whom live in low and middle income countries. In 2003, 3 million people worldwide with HIV/AIDS died. In the United States, nearly one million people are living with HIV/AIDS, and by the end of 2002, more than 500,000 people with HIV/AIDS had died. As shocking as these numbers are, they do not begin to adequately reflect the physical and emotional devastation to individuals, families, and communities coping with HIV/AIDS, nor do they capture the huge deleterious impact of HIV/AIDS on the economies and security of nations, and indeed entire regions. Even as the burden of HIV/AIDS continues to grow, recent developments provide some measure of optimism. For example four new antiretroviral drugs were licensed in 2003 by the U.S. Food and Drug Administration (FDA), each of which built on NIAID-sponsored research and/or has been tested in NIAID clinical trials networks. Many other "next-generation" anti-HIV drugs are in clinical trials.

A vaccine that prevents HIV infection -- or at least slows the progression of disease -- is a critical NIAID priority. Vaccine developers face formidable obstacles, including the genetic diversity of the virus and the lack of a clear understanding of the immune responses that might protect against HIV infection. Nonetheless, NIAID and our academic, industrial, international and philanthropic partners have made significant progress. Numerous HIV vaccine candidates are in various stages of preclinical and clinical development. The new Partnership for AIDS Vaccine Evaluation (PAVE) promises to optimize these efforts. PAVE is a coordinated HIV vaccine research effort that includes the three government agencies most involved in this activity -- NIH, the Centers for Disease Control and Prevention (CDC), and the Department of Defense. These agencies will work together to ensure that research protocols, standards, and measures are developed in a coordinated and harmonized manner so that outcomes can be compared across trials in the most cost effective and scientifically efficient manner. International non-government organizations (NGOs) and companies also have expressed interest in joining the partnership. Concurrently, novel approaches to HIV prevention are being studied and validated, including topically applied microbicides that individuals could use to protect themselves from HIV and other sexually transmitted pathogens. As discussed in the new NIAID Strategic Plan for Topical Microbicides, more than 50 candidate agents have shown laboratory activity against HIV and other STDs, and several of these agents have demonstrated safety and efficacy in animal models. In small human studies, several products have proven safe; later this year, NIAID's HIV Prevention Trials Network (HPTN), in conjunction with the National Institute of Child Health and Human Development, will launch a large international study to test two promising products in more than 3,000 women at high risk of acquiring HIV in the United States, five African countries, and India.

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Research on Other Emerging and Emerging Infectious Diseases

Infectious diseases have always afflicted humanity, and they will continue to confront us as long as man and microbes co-exist. Unfortunately, the viruses, bacteria, and parasites that cause infectious diseases do not remain static, but continually and dramatically change over time as new pathogens (such as HIV and the SARS coronavirus) emerge and as familiar ones (such as influenza virus and West Nile virus) re-emerge with new properties or in unfamiliar settings.

West Nile virus (WNV) first appeared in the western hemisphere in 1999, and by 2003 had spread to 45 states in the United States. NIAID has moved quickly to address this threat with basic research on the virus and its maintenance in nature, the development of vaccines and treatments, and the provision of reagents and other resources to the research community. NIAID also is supporting the development of three types of vaccines, as well as the screening and testing of WNV therapies. For example, the NIAID-sponsored Collaborative Antiviral Study Group is assessing the safety and efficacy of WNV immunoglobulins in patients with, or at high risk of serious brain diseases caused by WNV.

Severe acute respiratory syndrome (SARS) is a new infectious disease first identified in humans in early 2003. The prompt recognition that SARS is caused by a new type of coronavirus, and the rapid progress in SARS research reflect the dedication of and collaboration by the world's medical researchers and public health experts, including NIAID-sponsored scientists in the United States and abroad. NIAID supports research to understand the epidemiology and biology of the SARS virus and how it spreads, and to develop SARS countermeasures. Several approaches to SARS countermeasures are being pursued by the NIAID Laboratory of Infectious Diseases, the NIAID Vaccine Research Center, and by our contractors and grantees. For example, NIAID is participating in a project to screen up to 100,000 antiviral drugs and other compounds for activity against the SARS virus, and will test the most promising in animal models and human clinical trials. A number of compounds have shown promise in the test tube, including alpha interferon, a drug already approved by the FDA for the treatment of hepatitis B and C infections.

NIAID scientists and grantees are pursuing several parallel approaches in the search for a SARS vaccine. Once these experimental vaccines are ready, NIAID plans to test them in human clinical trials in our network of Vaccine and Treatment Evaluation Units. New research suggests that a SARS vaccine is within reach: NIAID intramural scientists have demonstrated that the mouse immune system develops antibodies capable of single-handedly neutralizing the SARS virus. This discovery affirms that researchers developing vaccines that trigger antibodies to the SARS virus are heading in the right direction. These findings also indicate that drug researchers can use laboratory mice as a model to evaluate whether a drug blocks the SARS virus. Both findings could help lessen the time it takes to develop an effective vaccine or antiviral drugs for SARS.

Influenza is a classic example of a re-emerging disease; it is not a new disease, but it continually changes. Because the replication machinery of the influenza virus is error prone, as the virus multiplies it can mutate to a slightly different form; this is referred to an "antigenic drift." Such viruses might require a slight modification of the yearly influenza vaccine to accommodate these changes. In addition, non-human influenza viruses such as avian influenza, can emerge that may be able to jump species into domestic poultry, farm animals such as pigs, and humans. This type of significant change in the antigenic makeup of the virus is referred to as "antigenic shift." Deadly pandemics associated with antigenic shifts are known to have occurred in 1918, 1957, and 1968. The pandemic that occurred in 1918-1919 after an antigenic shift killed 20-40 million people worldwide, including more than half a million in the United States. This recent history explains the current high level of concern about the appearance of new forms of virulent H5N1 avian influenza viruses in Asia that can adapt themselves by mutation to infect humans as has been the case already in dozens of individuals in Viet Nam and Thailand. Of even greater concern is the possibility that this avian virus can combine or reassort its genes with a human influenza virus and acquire the capability of readily spreading from person to person resulting in a new pandemic. Given the poor condition of public health systems in many underdeveloped regions and the speed of modern air travel, the consequences of such an event, should it result in an influenza pandemic, would be severe.

To address this threat, NIAID supports a broad program to develop more effective approaches to controlling influenza virus infections. Research includes programs to understand the pathogenesis, transmissibility, evolution, epidemiology, and the immune response to influenza viruses, as well as to develop new diagnostics, antiviral drugs and vaccines. NIAID currently supports several research projects to develop vaccines that could be manufactured more rapidly, are more broadly cross-protective, and are more effective than current influenza vaccines. The use of reverse genetics -- a tool developed by NIAID grantees -- holds the promise for more rapid generation of vaccine candidates that match the anticipated strain expected to circulate during the influenza season. Reverse genetics also can be used to turn highly pathogenic influenza viruses into vaccine candidates more suitable for vaccine manufacturing by removing or modifying certain virulence genes; laboratories around the world are using the technique to prepare vaccine candidates against the H5N1 viruses emerging in Asia. NIAID also is funding the development of new influenza vaccine technologies. Recently, NIAID supported a Phase II clinical trial of a new influenza vaccine produced in a cell culture system as an alternative to manufacturing the vaccine in eggs. Because NIAID has had remarkable success in the past with groundbreaking vaccine research -- including advances that led to hepatitis B, Haemophilus influenzae b, pneumoccocal pneumonia, and acellular pertussis vaccines - we are confident that one of the approaches that we are pursuing also will lead to a useful, "next-generation" influenza vaccine that can readily be adapted to emerging influenza strains.

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Research on Immune-Mediated Diseases

Immune-mediated diseases such as autoimmune diseases, allergic diseases, and asthma are important health challenges in the United States and abroad. Autoimmune diseases afflict 5 to 8 percent of the U.S. population; asthma and allergic diseases combined represent the sixth leading cause of chronic illness and disability in the United States, and the leading cause among children. The past two decades of fundamental research in immunology have resulted in a wealth of new information and extraordinary growth in our conceptual understanding of the immune system and the pathogenesis of immune-mediated diseases, which has led to the development of many useful therapies. For instance, we now have powerful treatments that selectively target several of the immune system molecules that cause inflammation, a hallmark of many autoimmune diseases. NIAID-sponsored researchers are now developing novel ways of selectively blocking inappropriate or destructive immune responses, while leaving protective immune responses intact, an area of research known as tolerance induction. In the Immune Tolerance Network, a consortium of basic and clinical scientists, promising studies are underway using tolerance induction to treat autoimmune diseases, such as rheumatoid arthritis, type 1 diabetes, and multiple sclerosis; asthma and allergic diseases; and the rejection of transplanted organs, tissues, and cells. So-called "tolerogenic" therapies would replace current lifelong non-specific immunosuppressive regimens (and their often debilitating side-effects) with short-term specific regimens that hold the promise of being curative.

Other important research is being conducted by the recently expanded Autoimmunity Centers of Excellence. The nine centers that make up this program conduct basic research and clinical trials on new immune-based therapies for diseases that collectively afflict between 14 and 22 million Americans. The Institute and our collaborators also have significantly bolstered the study of primary immunodeficiency diseases -- disorders caused by inherited flaws in the immune system that increase susceptibility to infections --with funding of the Primary Immunodeficiency Research Consortium (PIRC), a coalition of the world's most prominent researchers in the field of primary immunodeficiency diseases.

Another important NIAID research focus is the development of new interventions to reduce the burden of asthma, a significant and growing public health problem in the United States and many nations worldwide. NIAID has long been at the forefront of discoveries leading to the characterization of asthma and allergic diseases and is now vigorously pursing the translation of basic knowledge into more effective treatment and prevention strategies. To develop interventions to prevent the onset of asthma, more information is needed on the events that induce asthma. NIAID's Inner-City Asthma Consortium (ICAC) will soon launch a large study to define and analyze immunological and environmental influences upon the development of childhood asthma in a cohort of urban children followed from birth.

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Conclusion

With a strong research base, talented investigators in the United States and abroad, and the availability of powerful new research tools, NIAID anticipates that our basic and applied research programs will provide the countermeasures to improve our defenses against those who would attempt to harm us with bioterrorism, will develop new tools in the fights against HIV/AIDS and other infectious diseases, and will improve therapies and management of immune-mediated diseases.

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ANTHONY S. FAUCI, M.D.

Director, National Institute of Allergy and Infectious Diseases

National Institutes of Health

Dr. Anthony S. Fauci, a native of Brooklyn, New York, received his M.D. degree from Cornell University Medical College in 1966. He then completed an internship and residency at The New York Hospital-Cornell Medical Center. In 1968, Dr. Fauci came to the National Institutes of Health (NIH) as a clinical associate in the Laboratory of Clinical Investigation (LCI) at the National Institute of Allergy and Infectious Diseases (NIAID). In 1974, he became Head of the Clinical Physiology Section, LCI, and in 1980 was appointed Chief of the Laboratory of Immunoregulation, a position he still holds. In 1984, Dr. Fauci became Director of NIAID, where he oversees an extensive research portfolio of basic and applied research to prevent, diagnose, and treat infectious and immune-mediated illnesses, including HIV/AIDS and other sexually transmitted diseases, illness from potential agents of bioterrorism, tuberculosis, malaria, autoimmune disorders, asthma and allergies. Dr. Fauci serves as one of the key advisors to the White House and Department of Health and Human Services on global AIDS issues, and on initiatives to bolster medical and public health preparedness against possible future bioterrorist attacks.

Dr. Fauci has made many contributions to basic and clinical research on the pathogenesis and treatment of immune-mediated diseases. He has pioneered the field of human immunoregulation by making a number of basic scientific observations that serve as the basis for current understanding of the regulation of the human immune response. In addition, Dr. Fauci is widely recognized for delineating the precise mechanisms whereby immunosuppressive agents modulate the human immune response. He has developed effective therapies for formerly fatal diseases such as polyarteritis nodosa, Wegener's granulomatosis, and lymphomatoid granulomatosis. A 1985 Stanford University Arthritis Center Survey of the American Rheumatism Association membership ranked the work of Dr. Fauci on the treatment of polyarteritis nodosa and Wegener's granulomatosis as one of the most important advances in patient management in rheumatology over the previous 20 years.

Dr. Fauci has made seminal contributions to the understanding of how the AIDS virus destroys the body's defenses leading to its susceptibility to deadly infections. He also has delineated the mechanisms of induction of HIV expression by endogenous cytokines. Furthermore, he has been instrumental in developing strategies for the therapy and immune reconstitution of patients with this serious disease, as well as for a vaccine to prevent HIV infection. He continues to devote much of his research time to identifying the nature of the immunopathogenic mechanisms of HIV infection and the scope of the body's immune responses to the AIDS retrovirus.

In 2003, an Institute for Scientific Information study indicated that in the twenty year period from 1983 to 2002, Dr. Fauci was the 13th most-cited scientist among the 2.5 to 3 million authors in all disciplines throughout the world who published articles in scientific journals during that time frame. Dr. Fauci was the ninth most-cited scientist in the field of immunology in the period from January 1993 to June 30, 2003.

Through the years, Dr. Fauci has served as Visiting Professor at major medical centers throughout the country. He has delivered many major lectureships all over the world and is the recipient of numerous prestigious awards for his scientific accomplishments, including 28 honorary doctorate degrees from universities in the United States and abroad.

Dr. Fauci is a member of the National Academy of Sciences, the American Philosophical Society, the Institute of Medicine of the National Academy of Sciences (Council Member), the American Academy of Arts and Sciences, and the Royal Danish Academy of Science and Letters, as well as a number of other professional societies including the American College of Physicians, the American Society for Clinical Investigation, the Association of American Physicians, the Infectious Diseases Society of America, the American Association of Immunologists, and the American Academy of Allergy Asthma and Immunology. He serves on the editorial boards of many scientific journals; as an editor of Harrison's Principles of Internal Medicine; and as author, coauthor, or editor of more than 1,000 scientific publications, including several textbooks.

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Department of Health and Human Services

Office of Budget

William R. Beldon

Mr. Beldon is currently serving as Acting Deputy Assistant Secretary for Budget, HHS. He has been a Division Director in the Budget Office for 16 years, most recently as Director of the Division of Discretionary Programs. Mr. Beldon started in federal service as an auditor in the Health, Education and Welfare Financial Management Intern program. Over the course of 30 years in the Budget Office, Mr. Beldon has held Program Analyst, Branch Chief and Division Director positions. Mr. Beldon received a Bachelor's Degree in History and Political Science from Marshall University and attended the University of Pittsburgh where he studied Public Administration. He resides in Fort Washington, Maryland.

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Last Updated April 01, 2004